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Good day! Child KJ is again house! Additionally, we focus on the questionable efficacy of ctDNA blood exams, see what Vinay Prasad has to say at NORD, and extra.
After groundbreaking CRISPR remedy, a homecoming
From my colleague Jason Mast: KJ Muldoon, the primary toddler handled with a personalised gene-editing drug, has been discharged from the hospital for the primary time in his life, the Kids’s Hospital of Philadelphia introduced Tuesday. After 307 days at CHOP, KJ was wearing what early research counsel are the world’s smallest and cutest commencement cap-and-gown earlier than leaving. 
It’s a promising signal for KJ, his household, and the remedy researchers in-built simply 6 months. Born with an ultra-rare illness that prevented his liver from breaking down ammonia, he was rushed to CHOP, the place specialists saved him beneath strict monitoring to stop ammonia from flaring and doing long-term harm. Sometimes, he would obtain a liver transplant. As a substitute, scientists throughout the nation teamed to plan a gene-editing remedy.
KJ obtained the primary of three doses at six months previous. He’s now 10 months previous. The hope is that, whereas not cured, he has been left with a much more gentle model of his illness. In the meantime, researchers are engaged on methods to scale his remedy for 1000’s of others.
ctDNA testing surges, however affect stays unproven
Circulating tumor DNA blood exams are quickly gaining recognition amongst oncologists for his or her potential to detect most cancers recurrence and remedy resistance sooner than conventional imaging. However regardless of their promise, information introduced on the annual ASCO assembly this week counsel that the scientific worth of ctDNA exams continues to be unproven.
Whereas some trials trace at advantages, others lack conclusive survival outcomes — which raises concern about whether or not early interventions based mostly on ctDNA truly enhance sufferers’ lives.
“We’d like proof to know the true worth of those exams,” College of Pennsylvania most cancers researcher Angela DeMichelle advised STAT’s Angus Chen. “They’ve unbelievable potential to assist individuals, but when we don’t do the research and don’t do them the precise manner, we are able to damage individuals.”
Vinay Prasad talks RCTs, endpoints at NORD
Vinay Prasad, the brand new chief of CBER on the FDA, spoke at a gathering of the Nationwide Group for Uncommon Problems yesterday. Although for years the firebrand oncologist emphasised the significance of rigorous randomized managed trials, he now asserts that not each drug for each illness wants one — particularly ultra-rare illnesses. He cited the case of Child KJ, saying “the U.S. FDA is firmly dedicated to creating positive all of those therapies come by means of as rapidly as posisble, and that individuals that individuals have entry to those therapies as quickly as attainable.”
He additionally appeared to strike a unique tune on one other situation for which he has demonstrated zeal: “Some individuals might imagine I’m a critic of surrogate endpoints and I solely need total survival. However I need to say the reply is each,” he mentioned, including that the FDA “will take motion on the first signal of promise for uncommon illnesses.”
The company’s method, he mentioned, is to “take motion on the earliest statistically persuasive biomarker modifications which might be fairly prone to predict scientific outcomes.”
Opinion: Change to Covid vaccine suggestions may jeopardize pregnant well being
HHS Secretary Robert F. Kennedy, Jr.’s transfer to drop Covid vaccine suggestion for pregnant individuals represents a serious moral and public well being setback, opines Johns Hopkins bioethicist Ruth Faden.
In a brand new First Opinion, she warns that reversing course on vaccination steerage ignores clear proof of the vaccine’s advantages for each moms and their newborns — safety that’s particularly essential within the face of heightened dangers like preterm start and stillbirth.
Faden, who helped lead nationwide efforts to shut the longstanding medical analysis hole round being pregnant in pandemics, says Kennedy’s push to require randomized managed trials is each scientifically pointless and ethically indefensible.
